Lin Ming-Hao, Wang Ya-Jun, Zhang Yuan-Yuan, Cheng Yi-Fei, Sun Yu-Qian, Mo Xiao-Dong, Yan Chen-Hua, Chen Yu-Hong, Chen Yao, Wang Jing-Zhi, Wang Feng-Rong, Han Ting-Ting, Han Wei, Chen Huan, Wang Yu, Zhang Xiao-Hui, Huang Xiao-Jun, Xu Lan-Ping, Xu Zheng-Li
Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, Peking University, Beijing, China.
First Hospital of Shanxi Medical University, Taiyuan, China.
Ann Hematol. 2025 Jun 4. doi: 10.1007/s00277-025-06440-9.
This study aimed to evaluate the outcomes of second haploidentical hematopoietic stem cell transplantation (HID-HSCT) for patients with severe aplastic anaemia (SAA) who experienced graft failure. Twenty-one SAA patients with graft failure after first allogeneic (allo-) HSCT were enrolled, including 6 (28.6%) with poor graft function and 15 (71.4%) with graft rejection. Two patients developed primary graft failure after the second transplantation, and the cumulative incidence of neutrophil recovery was 81.0% ± 0.9%, with a median recovery of 13 days (range, 10-23 days). The cumulative incidences of grade II-IV and III-IV acute graft-versus-host disease (GvHD) were 19.0% ± 0.8% and 4.8% ± 0.2%, respectively. The cumulative incidences of chronic GvHD (cGvHD) at three and five years were both 24.5% ± 1.0%, with no severe cGvHD cases. The 5-year overall survival (OS) rate was 47.1% ± 11.0%, with a median follow-up of 2037.5 days (range, 863-3488 days) among survivors. Multivariate logistic regression analysis identified not changing a donor (P = 0.019) as the only adverse factor for graft failure after the second HID-HSCT. Moreover, changing a donor (P = 0.004), compatible donor-recipient blood type (P = 0.005), and patient age < 12 years (P = 0.038) at the second transplantation were significant predictors of improved OS. These findings suggest that a second HID-HSCT could be a feasible option for treating graft failure in SAA patients.
本研究旨在评估第二次单倍型相合造血干细胞移植(HID-HSCT)对经历移植物失败的重型再生障碍性贫血(SAA)患者的疗效。纳入了21例首次异基因造血干细胞移植(allo-HSCT)后发生移植物失败的SAA患者,其中6例(28.6%)移植物功能不良,15例(71.4%)发生移植物排斥。2例患者在第二次移植后发生原发性移植物失败,中性粒细胞恢复的累积发生率为81.0%±0.9%,中位恢复时间为13天(范围10-23天)。II-IV级和III-IV级急性移植物抗宿主病(GvHD)的累积发生率分别为19.0%±0.8%和4.8%±0.2%。慢性GvHD(cGvHD)在3年和5年时的累积发生率均为24.5%±1.0%,无严重cGvHD病例。5年总生存率(OS)为47.1%±11.0%,幸存者的中位随访时间为2037.5天(范围863-3488天)。多因素逻辑回归分析确定在第二次HID-HSCT后不更换供者(P = 0.019)是移植物失败的唯一不利因素。此外,在第二次移植时更换供者(P = 0.004)、供受者血型相合(P = 0.005)以及患者年龄<12岁(P = 0.038)是OS改善的显著预测因素。这些发现表明,第二次HID-HSCT可能是治疗SAA患者移植物失败的一种可行选择。